The molecular mechanisms of transient leukemia by GATA1 mutation and development of molecular target therapy

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We performed this study to understand the role of GATA1 mutations in the development of transient leukemia and develop the molecular target therapy, and found the following results.1. We found the essential role of SCF/KIT signaling in the proliferation of transient leukemia in Down syndrome.2. We showed that the mutations in the GATA1 genes affect the expression levels of GATA1s protein. Furthermore, GATA1s low mutations were significantly associated with a risk of progression to ML-DS and lower WBC counts.